The TRND program at the National Center for Advancing Translational Sciences (NCATSjNIH) was established in 2009 to stimulate and speed the discovery & development of new drugs for rare and neglected diseases. It is a unique collaborative model between the NIH and academic scientists, non- profit organizations and pharmaceutical and biotechnology companies. These collaborations involve early drug discovery through high Throughput Screening (HTS) and repurposing known drugs to the clinic via access to development capabilities, such as lead optimization, ADME, PKjPD, toxicology, formulation development, scale up API and clinical & regulatory activities.
The involvement of RAS pathways in many rare diseases (RASopathies) is well recognized, and the current symposium provides an excellent venue to discuss potential collaboration and non-traditional funding models available at NCATS to rapidly advance therapeutic innovations directly to the patients with RASopathies. The current TRND portfolio and case studies on rare disease drug development will be highlighted to exemplify potential opport ities for collaboration.